Targeted therapy for brain tumors: overview and application to precision medicine

Targeted cancer therapies for cancer treatment are at the forefront of cancer treatments. The identification of pathologic cell signaling pathways in cancer cells via precision medicine enables the development of new therapies to target the underpinning molecular changes within tumor cells. Applying these techniques to brain tumors is no different, except for the added necessity that these therapeutics penetrate the blood-brain barrier. Besides cell signaling alterations seen in other cancers outside of the brain (such as receptor tyrosine kinase pathways, Raf/MEK/Erk pathway, PI3K/AKT/mTOR pathway, to name a few), there are several cell-signaling pathway aberrations unique to central nervous system tumors (for example isocitrate dehydrogenase mutations or O6-methylguanine DNA methyltransferase methylation status) that allow for therapies to specifically target brain tumors. Herein, we describe the various molecular pathway aberrations that have been identified and used to develop targeted molecular therapies for brain tumors.